Gannex Publishes Phase I Data of ASC42, a Novel Farnesoid X Receptor Agonist in the Journal Drugs in R&D

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Gannex Publishes Phase I Data of ASC42, a Novel Farnesoid X Receptor Agonist in the Journal Drugs in R&D

Gannex Pharma Co., Ltd. (“Gannex”), a wholly-owned company of Ascletis Pharma Inc. (HKEX: 1672) has announced the publication of safety, pharmacokinetics (PK), and pharmacodynamics (PD) data of ASC42, a groundbreaking farnesoid X receptor (FXR) agonist, in healthy subjects.

This milestone has been achieved in the prestigious journal, Drugs in R&D.

ASC42: A Novel FXR Agonist

Developed in-house by Gannex, ASC42 represents a non-steroidal, selective, potent FXR agonist with remarkable potential and a robust global intellectual property portfolio.

Phase I Study Results

The published study demonstrates that ASC42 exhibits a generally safe and well-tolerated profile. The following key findings were noted:

• ASC42 demonstrated safety when administered as single doses up to 100 mg and as multiple daily doses for 14 days at a maximum of 15 mg in healthy subjects.

• At the therapeutic dose range of 5 mg to 15 mg, ASC42 displayed an acceptable safety profile, with no instances of drug-induced pruritus or transient elevations in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), or γ-glutamyl transferase (GGT).

• ASC42 exhibited effective FXR target engagement, leading to dose-dependent elevations in Fibroblast Growth Factor 19 (FGF19) and a reduction in 7α-hydroxy-4-cholesten-3-one (C4) levels.

• No significant changes in total cholesterol (TC), high-density lipoprotein cholesterol (HDL-C), and low-density lipoprotein cholesterol (LDL-C) were observed in healthy subjects following daily dosing for 14 days.

These encouraging results support the continued exploration of ASC42 in patients with non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and primary biliary cholangitis (PBC).

Addressing a Critical Medical Need

Notably, an epidemiology study highlighted the substantial unmet clinical need for PBC treatment in China, with approximately 656,000 PBC patients, including 440,000 females over the age of 40. Currently, Ursodeoxycholic acid (UDCA) is the sole approved treatment for PBC in China, with around 40% of patients showing an inadequate response or intolerance to UDCA. Obeticholic Acid (OCA), an approved medicine in the U.S. for PBC patients who do not respond adequately to UDCA, presents significant side effects, including increased pruritus rates and elevated LDL-C levels.

Gannex’s ongoing Phase II clinical trial for ASC42 in PBC, with patient enrollment recently completed as of July 20, 2023, holds the promise of addressing this critical unmet clinical need in China.

Dr. Jinzi J. Wu’s Insights

Dr. Jinzi J. Wu, Founder, Chairman, and CEO of Ascletis, emphasized, “PBC remains to be a huge unmet clinical need in China, and the current treatment for patients is very limited. The Phase II clinical trial of Gannex’s in-house developed farnesoid X receptor agonist ASC42 will be completed soon. The topline data of the Phase II clinical trial is expected to be released by the end of 2023.”

About Ascletis

Ascletis is an innovative biotech company listed on the Hong Kong Stock Exchange (1672.HK), with a focus on research and development. Covering the entire value chain from discovery and development to manufacturing and commercialization, Ascletis specializes in three therapeutic areas with global unmet medical needs: viral diseases, non-alcoholic steatohepatitis (NASH), and oncology. The company’s management team boasts deep expertise and a successful track record. Ascletis is committed to rapidly advancing its drug pipeline to compete globally, with multiple drug candidates in various stages of development. Notable candidates include ASC22 (CHB functional cure), ASC40 (acne), ASC40 (recurrent glioblastoma), ASC40 (NASH), ASC41 (NASH), and ASC61 (advanced solid tumors).

Source: Ascletis Pharma Inc.